RU2017120359A - INDUCTION OF OSTEOGENESIS BY INTRODUCING RNA CODING BONE MORPHOGENETIC PROTEIN (KMB) - Google Patents

INDUCTION OF OSTEOGENESIS BY INTRODUCING RNA CODING BONE MORPHOGENETIC PROTEIN (KMB) Download PDF

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RU2017120359A
RU2017120359A RU2017120359A RU2017120359A RU2017120359A RU 2017120359 A RU2017120359 A RU 2017120359A RU 2017120359 A RU2017120359 A RU 2017120359A RU 2017120359 A RU2017120359 A RU 2017120359A RU 2017120359 A RU2017120359 A RU 2017120359A
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pharmaceutical composition
rna
cells
matrix
bone
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RU2017120359A
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Russian (ru)
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RU2718590C2 (en
RU2017120359A3 (en
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Элизабет БАЛЬМАЙОР
Карстен Рудольф
Кристиан Планк
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Этрис Гмбх
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/33—Fibroblasts
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease

Claims (26)

1. Фармацевтическая композиция, включающая полирибонуклеотид (РНК) с последовательностью, которая кодирует костный морфогенетический белок (КМБ), для применения в:1. A pharmaceutical composition comprising a polyribonucleotide (RNA) with a sequence that encodes a bone morphogenetic protein (KMB), for use in: (i) лечении или предупреждении костного заболевания, костного расстройства или костного повреждения, и/или(i) treating or preventing bone disease, bone disorder or bone damage, and / or (ii) индукции или повышении остеогенной дифференциации, остеогенеза, окостенения, костной регенерации и/или костного морфогенеза,(ii) the induction or increase of osteogenic differentiation, osteogenesis, ossification, bone regeneration and / or bone morphogenesis, у пациента.in a patient. 2. Фармацевтическая композиция для применения по п. 1, в которой КМБ является КМБ-2 или КМБ-7.2. The pharmaceutical composition for use according to claim 1, in which KMB is KMB-2 or KMB-7. 3. Фармацевтическая композиция для применения по пп. 1-2, в которой РНК не является химически модифицированной РНК или является химически модифицированной РНК.3. The pharmaceutical composition for use in PP. 1-2, in which the RNA is not chemically modified RNA or is chemically modified RNA. 4. Фармацевтическая композиция для применения по пп. 1-3, дополнительно включающая один или несколько агентов для доставки и/или интродукции указанной РНК в клетки-мишени или в ткани-мишени.4. The pharmaceutical composition for use in PP. 1-3, further comprising one or more agents for the delivery and / or introduction of said RNA into target cells or target tissue. 5. Фармацевтическая композиция для применения по пп. 1-3, дополнительно включающая реагент для липосомальной трансфекции (РЛТ).5. The pharmaceutical composition for use in PP. 1-3, further comprising a reagent for liposomal transfection (RLT). 6. Фармацевтическая композиция для применения по пп. 4-5, в которой указанная РНК формирует комплекс с указанным агентом, реагентом или РЛТ.6. The pharmaceutical composition for use in PP. 4-5, in which the specified RNA forms a complex with the specified agent, reagent or RLT. 7. Фармацевтическая композиция для применения по пп. 1-6, в которых указанная РНК доставляют in vivo.7. The pharmaceutical composition for use in PP. 1-6, wherein said RNA is delivered in vivo. 8. Фармацевтическая композиция для применения по пп. 1-6, в которых указанную РНК доставляют ex vivo в клетки, которые интродуцируют указанному пациенту.8. The pharmaceutical composition for use in PP. 1-6, in which the specified RNA is delivered ex vivo to cells that are introduced to the specified patient. 9. Фармацевтическая композиция для применения по п. 8, в котором указанную РНК доставляют ex vivo в клетки пациента, и в котором клетки, в которые доставляют РНК, реинтродуцируют указанному пациенту.9. The pharmaceutical composition for use according to claim 8, wherein said RNA is delivered ex vivo to the patient’s cells, and in which the cells to which the RNA is delivered are reintroduced to said patient. 10. Фармацевтическая композиция для применения по пп. 1-9, в которых указанную РНК и/или указанные клетки предназначены для введения в ткань или в непосредственной близости к ткани пациента, в которой индукция роста костей желательна.10. The pharmaceutical composition for use in PP. 1-9, in which the specified RNA and / or these cells are intended for introduction into the tissue or in close proximity to the tissue of the patient, in which the induction of bone growth is desirable. 11. Фармацевтическая композиция для применения по пп. 8-10, в которых указанные клетки являются остеопрогениторными клетками.11. The pharmaceutical composition for use in PP. 8-10, in which these cells are osteoprogenic cells. 12. Фармацевтическая композиция для применения по пп. 8-11, в которых указанные клетки являются мезенхимальными стволовыми клетками (МСК).12. The pharmaceutical composition for use in PP. 8-11, in which these cells are mesenchymal stem cells (MSCs). 13. Фармацевтическая композиция для применения по п. 12, в котором указанные МСК являются мезенхимальными стволовыми клетками, производными жировой ткани (ЖМСК), или мезенхимальными стволовыми клетками, производными костного мозга (КММСК).13. The pharmaceutical composition for use according to claim 12, wherein said MSCs are mesenchymal stem cells derived from adipose tissue (JMSC) or mesenchymal stem cells derived from bone marrow (CMSC). 14. Фармацевтическая композиция для применения по пп. 1-13, дополнительно включающая матрикс или каркас, к которым добавляют указанную РНК или в которую нагружают указанной РНК.14. The pharmaceutical composition for use in PP. 1-13, further comprising a matrix or framework to which the indicated RNA is added or to which the indicated RNA is loaded. 15. Фармацевтическая композиция для применения по п. 14, в которой матрикс или каркас включает коллаген и/или фибрин.15. The pharmaceutical composition for use according to claim 14, in which the matrix or framework includes collagen and / or fibrin. 16. Фармацевтическая композиция для применения по пп. 14-15, в которых матрикс или каркас является коллагеновой губкой и/или сгустком фибрина, или фибриновым клеем.16. The pharmaceutical composition for use according to claims. 14-15, in which the matrix or framework is a collagen sponge and / or clot of fibrin, or fibrin glue. 17. Фармацевтическая композиция для применения по пп. 14-16, в которых матрикс или каркас является высушенным в вакууме.17. The pharmaceutical composition for use in PP. 14-16, in which the matrix or framework is dried in vacuum. 18. Фармацевтическая композиция для применения по пп. 8-13, в которых указанные клетки высевают на матрикс или каркас в соответствии с одним из пп. 14-17.18. The pharmaceutical composition for use in PP. 8-13, in which these cells are seeded on a matrix or scaffold in accordance with one of paragraphs. 14-17. 19. Фармацевтическая композиция для применения по пп. 14-18, в которых указанный матрикс или каркас трансплантируют в кость или костную ткань указанного пациента.19. The pharmaceutical composition for use in PP. 14-18, wherein said matrix or scaffold is transplanted into a bone or bone tissue of said patient. 20. Фармацевтическая композиция для применения по пп. 3-19, в которой 25% цитидинов указанной химически модифицированной РНК являются 5-метилцитидинами (m5С) и 25% уридинов указанной химически модифицированной РНК являются 2-тиоуридинами (s2U).20. The pharmaceutical composition for use in PP. 3-19, in which 25% of the cytidines of the indicated chemically modified RNA are 5-methylcytidines (m5C) and 25% of the uridines of the indicated chemically modified RNA are 2-thiouridines (s2U). 21. Матрикс или каркас в соответствии с одним пп. 14-20.21. Matrix or frame in accordance with one paragraph. 14-20. 22. Фармацевтическая композиция, включающая матрикс или каркас по п. 21.22. A pharmaceutical composition comprising a matrix or framework according to claim 21. 23. Фармацевтическая композиция по пп. 1-20, матрикс или каркас по п. 21 или фармацевтическая композиция по п. 22, которые перерабатывают и/или предусматривают применять для устойчивой и/или замедленной доставки указанной РНК.23. The pharmaceutical composition according to paragraphs. 1-20, the matrix or framework according to claim 21 or the pharmaceutical composition according to claim 22, which are processed and / or contemplated to be used for stable and / or delayed delivery of said RNA.
RU2017120359A 2014-11-10 2015-11-10 Induction of osteogenesis by introduction of rna coding bone morphogenetic protein (bmp) RU2718590C2 (en)

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EP14192539 2014-11-10
EP14192539.6 2014-11-10
PCT/EP2015/076238 WO2016075154A1 (en) 2014-11-10 2015-11-10 Induction of osteogenesis by delivering bmp encoding rna

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RU2017120359A3 RU2017120359A3 (en) 2019-05-23
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JP (2) JP2017534649A (en)
KR (1) KR102647743B1 (en)
CN (2) CN113786498A (en)
AU (1) AU2015345164B2 (en)
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CA (1) CA2966836A1 (en)
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BR112017009505A2 (en) 2018-02-06
EP3218012A1 (en) 2017-09-20
AU2015345164B2 (en) 2021-05-06
CN107405412A (en) 2017-11-28
JP2021185168A (en) 2021-12-09
KR102647743B1 (en) 2024-03-14
JP2017534649A (en) 2017-11-24
JP7373528B2 (en) 2023-11-02
CN113786498A (en) 2021-12-14
KR20170081684A (en) 2017-07-12
RU2718590C2 (en) 2020-04-08
CA2966836A1 (en) 2016-05-19
US20180214572A1 (en) 2018-08-02
AU2015345164A1 (en) 2017-05-18
WO2016075154A1 (en) 2016-05-19
EP3542825A1 (en) 2019-09-25
RU2017120359A3 (en) 2019-05-23

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